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A myeloma drug given accelerated approval in 2021 and withdrawn in the US months later when its confirmatory trial suggested it shortened survival.
Melflufen is a lipophilic peptide-conjugated alkylator activated by aminopeptidases enriched in myeloma cells. Accelerated approval (February 2021) rested on the single-arm HORIZON response rate. The confirmatory OCEAN trial met its PFS endpoint but showed an overall survival detriment in a subgroup (patients with prior autologous transplant), leading to a partial clinical hold, an ODAC vote against, and US withdrawal (2021; formal FDA withdrawal 2024). It remains approved in the EU with restrictions.
Lesson: accelerated approval on response rate carries real risk when the confirmatory trial shows harm; the same subgroup could be a true signal or noise, and regulators in the US and EU read it differently.
Peptidase-activated alkylating prodrug of melphalan.
1.Melphalan flufenamide's antibody arm binds its target on the surface of the cancer cell.
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In combination with dexamethasone for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody
Accelerated approval on a surrogate endpoint, with a confirmatory trial required. source
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody
Withdrawn: the indication came off the label 3.0 years after its accelerated approval. source
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Query for this drug: (TITLE:"Melphalan flufenamide" OR ABSTRACT:"Melphalan flufenamide" OR TITLE:"Pepaxto" OR ABSTRACT:"Pepaxto" OR TITLE:"melflufen" OR ABSTRACT:"melflufen") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Melphalan flufenamide, not a curated reading list.
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